Arrowhead Pharmaceuticals has submitted an application seeking regulatory clearance to commence a Phase I/IIa clinical trial of its investigational RNA interference (RNAi) therapeutic, ARO-DM1, to potentially treat type 1 myotonic dystrophy (DM1).

DM1 is the most common adult-onset muscular dystrophy.

The company filed the application with a local Ethics Committee, as well as the New Zealand Medicines and Medical Devices Safety Authority, to be reviewed by the Standing Committee on Therapeutic Trials. 

Subject to regulatory clearance, the company will commence the dose-escalating trial of ARO-DM1 in up to 48 DM1 patients.

Dubbed ARODM1-1001, the study will assess the tolerability, safety, pharmacodynamics, and pharmacokinetics of ARO-DM1.

The therapy is claimed to reduce the dystrophia myotonica protein kinase (DMPK) gene expression. 

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At present, there exists no disease-modifying therapy approved for DM1.

Arrowhead discovery and translational medicine chief James Hamilton said: “ARO-DM1 is Arrowhead’s second clinical candidate utilising our TRiM platform to deliver RNAi therapeutics to skeletal muscle. 

“Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities, and may become physically disabled and have a shortened life expectancy. 

“ARO-DM1 represents a novel approach to treat DM1 by silencing aberrantly transcribed DMPK mRNA, which could lead to improvements in multiple symptoms, including muscle strength and function.”

In June 2023, the company submitted an application seeking clearance to begin a Phase I trial of ARO-SOD1 for the treatment of amyotrophic lateral sclerosis (ALS) harbouring superoxide dismutase 1 (SOD1) mutations in up to 24 participants.

This filing was made to an ethics committee, in accordance with the Australian Department of Health and Ageing Therapeutic Goods Administration’s clinical trial notification process.

The placebo-controlled, dose escalation, randomised trial was designed to analyse the safety and tolerability of ARO-SOD1 in adult ALS patients with SOD1 mutations.

The trial will also assess the pharmacodynamics and pharmacokinetics of the RNAi-based investigational medicine.

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

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